Burjeel Holdings and BridgeBio Pharma, Inc. have announced a new project to revolutionize the field of early diagnosis and treatment of rare diseases or disorders in the UAE and the region. The two entities signed a preliminary, non-binding Collaboration Agreement establishing a mutual intention to work together on project ‘NADER’ (Needs Assessment and Therapeutics Development for Rare Diseases – ‘nader’ meaning ‘rare’ in Arabic). Genetic in origin, these often life-threatening or chronically debilitating diseases affect a small percentage of the population and are present throughout the person's entire life, even if symptoms do not immediately appear.

The partnership will launch operations in Abu Dhabi to conduct clinical trials and research, leveraging the Emirate’s advanced infrastructure for innovation and life science. Project ‘NADER’ aims to revolutionize the field of early diagnosis and treatment in order to potentially improve patient outcomes. The entities intend to collaborate on identifying patients with several rare diseases, including achondroplasia, hypochondroplasia, FGFR-driven craniosynostoses, autosomal dominant hypocalcemia type 1, limb girdle muscular dystrophy type 2i, congenital adrenal hyperplasia, Canavan disease, propionic acidemia, methylmalonic acidemia, pantothenate kinase-associated neurodegeneration, and others as mutually agreed.

Even though these rare diseases affect a small percentage of the population and can present with nonspecific signs and symptoms, they are often misdiagnosed or only diagnosed later in the disease course after severe complications manifest. Accurate and early diagnosis are crucial to starting intervention promptly and avoiding disease progression. The project will also allow identification of patients who have already been diagnosed but do not currently have access to cutting edge therapies or clinical trial options.

The first phase will aim to set the roadmap to providing novel treatment options for identified patients through clinical trials.